FDA Okays First Treatment for Rare Thyroid Cancer

WASHINGTON -- The FDA has approved vandetanib for treatment of metastatic medullary thyroid cancer in patients with growing or symptomatic disease who are ineligible for surgery.

Drug approval was based on an international study of 331 patients in a metastatic phase of the rare cancer. Patients were randomized to drug treatment or placebo to an endpoint of progression-free survival.

Those in the treatment group had a longer period of progression-free survival when compared against placebo -- a median 22.6 months versus 16.4 months.

The study did not determine overall survival of the patients in the treatment group, an FDA statement said.

Adverse events included diarrhea, rash, nausea, hypertension, headache, fatigue, decreased appetite, abdominal pain, breathing complications, heart failure, and sepsis. Those latter three adverse events resulted in the deaths of five patients in the treatment group of the study, the statement said.

The drug also may cause irregular heartbeats, which can lead to death.

Vandetanib was approved with a risk evaluation and mitigation strategy (REMS) that requires healthcare professionals to inform patients of the potential heart-related risks the drug poses and to be certified through the REMS program before the drug can be prescribed or dispensed, the statement said.

The drug is manufactured by AstraZeneca and has not yet been given a trade name. There are currently no other FDA-approved treatments for this type of cancer.

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